On March 2, 2020 UroGen Pharma Ltd. (Nasdaq:URGN) a biopharmaceutical company dedicated to building novel solutions that treat specialty cancers and urologic diseases because patients deserve better options, reported financial results for the fourth quarter and full year ended December 31, 2019 and provided an overview of the Company’s recent developments (Press release, UroGen Pharma, MAR 2, 2020, View Source [SID1234555067]).

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"At UroGen, we are eagerly awaiting potential approval of our lead product candidate, UGN-101, for the treatment of patients with low-grade upper tract urothelial cancer (LG UTUC). The significant progress on key clinical, regulatory and commercial milestones in 2019 places us in a position of strength as we prepare to deliver the first non-surgical therapy for the treatment of LG UTUC. Our experienced commercial team has been working tirelessly to ensure we are prepared for launch, and we look forward to providing these patients with a new treatment option," said Liz Barrett, President and Chief Executive Officer (CEO) of UroGen. "We are also advancing multiple pipeline candidates in development for areas of unmet need in both low-grade and high-grade bladder cancer, including UGN-102 and UGN-302, respectively. As our team pioneers new treatments to improve patient care in specialty cancers and urologic diseases, we look forward to maximizing patient and shareholder value through the exciting events on the horizon".

2019 and Recent Highlights

UGN-101 (mitomycin gel) for instillation for Patients with LG UTUC Progress

Reported positive updated durability and complete response data from the pivotal Phase 3 OLYMPUS trial in September 2019. The data were consistent with previously reported results in May 2019 and January 2019.
Completed a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA).
Received FDA filing acceptance and Priority Review of the NDA for UGN-101, with a PDUFA goal date of April 18, 2020. If approved, UGN-101 will be the first non-surgical, chemoablative treatment option for LG UTUC.
UGN-101 Commercial Readiness

Hired an internal team with a track record of success in urology and oncology as well as a veteran sales force leadership team with deep uro-oncology relationships.
Implemented innovative solutions around patient identification, reimbursement and seamless logistics to enhance physician adoption upon launch. Based on recent market research, 88% of urologists desire a new and differentiated treatment option for their patients.
Executed agreements with 3PL, a specialty distributor and a national partner to provide prepared admixture to urology clinics.
Pipeline Expansion and Developments

UGN-102 (mitomycin gel) for intravesical instillation for patients with low-grade intermediate risk non-muscle invasive bladder cancer (LG IR NMIBC)
Completed enrollment ahead of schedule and reported positive interim results from the single-arm, open-label Phase 2b OPTIMA II trial of investigational UGN-102 for patients with LG IR NMIBC.
LG IR NMIBC is defined as those patients with one or two of the following criteria: multifocal disease, large tumors and rapid rates of recurrence. This is a patient population whereby the current standard of care, transurethral resection of bladder tumor, or TURBT, is used repeatedly to address chronic recurrence of disease. These patients experience what can be viewed as a form of surgical failure and many undergo multiple surgical procedures during life to "manage" bladder cancer recurrences.
There are no drugs currently approved by the FDA as first-line treatment for LG IR NMIBC. UGN-102 has the potential to provide a non-surgical treatment alternative for approximately 80,000 patients diagnosed with LG IR NMIBC.
UGN-201 (TLR7/8 agonist) for patients with high-grade non muscle invasive bladder cancer (HG NMIBC)
Shared nonclinical data of investigational UGN-201 (a TLR7/8 agonist) as a monotherapy and in combination with checkpoint inhibitors.
In murine models, UGN-201 in combination with local anti-CTLA-4 increased survival.
Entered into an exclusive worldwide license agreement with Agenus Inc. to develop and commercialize zalifrelimab (AGEN1884, anti-CTLA-4 antibody) via intravesical delivery in combination with UGN-201 for the treatment of high-grade urinary tract cancers, initially targeting HG NMIBC. The combination of UGN-201 and zalifrelimab is referred to as investigational agent UGN-302.
Corporate Achievements

Strengthened the Company’s financial position with a follow-on offering of approximately $162 million in January 2019.
2020 Anticipated Milestones and Product Development Plans

UGN-101

Publication of the final results of the primary endpoint from the OLYMPUS trial in patients with LG UTUC in 1H
UGN-101 potential approval and launch in Q2
UGN-102

Updated durability and complete response data from UGN-102 Phase 2b Study
Initiation of pivotal Phase 3 Study in 2H
UGN-302

Advancement to first in human clinical study following formulation and dose optimization
Fourth Quarter and Full Year 2019 Financial Results; 2020 Guidance

As of December 31, 2019, cash, cash equivalents and marketable securities totaled $195.6 million, excluding restricted cash.
Research and development expenses for the three months ended December 31, 2019 were $20.1 million, including non-cash share-based compensation expense of $1.9 million. Research and development expenses for the year ended December 31, 2019 were $49.3 million, including non-cash share-based compensation expense of $8.3 million. The research and development expenses for the three months and year ended December 31, 2019 included an in-process research and development charge of $10.0 million associated with the execution of the Agenus licensing agreement.
General and administrative expenses for the three months ended December 31, 2019 were $19.7 million, including non-cash share-based compensation expense of $6.2 million. General and administrative expenses for the year ended December 31, 2019 were $60.2 million, including non-cash share-based compensation expense of $21.7 million.
UroGen reported a net loss of $39.0 million, or basic and diluted net loss per ordinary share of $1.86, for the three months ended December 31, 2019. The Company reported a net loss of $105.1 million, or basic and diluted net loss per ordinary share of $5.12, for the year ended December 31, 2019.
The Company anticipates operating expenses in the range of $145 to $155 million for 2020. Non-cash stock-based compensation expense for 2020 is expected to be in the range of $32 to $36 million subject to market conditions, and other non-operating income for 2020 is anticipated to be approximately $2.5 million.
UroGen has 21.0 million ordinary shares outstanding.
Conference Call & Webcast Information

Members of UroGen’s management team will host a live conference call and webcast today at 8:30 AM Eastern Time to review the Company’s financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. Alternatively, please call (888) 771-4371 (U.S.) or (847) 585-4405 (International) to listen to the live conference call. The conference ID number for the live call will be 49393633. An archive of the webcast will be available for two weeks on the Company’s website.

On March 2, 2020 Medivir AB (Nasdaq Stockholm: MVIR) reported that new data from the completed phase Ia study with MIV-818 will be presented at the R&D day hosted by the company today, from 2 pm – 4.30 pm (CET) (Press release, Medivir, MAR 2, 2020, View Source [SID1234555083]).

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The primary objective of the phase Ia study was to evaluate safety and tolerability of MIV-818 in liver cancer patients. A total of nine patients with advanced disease were included: six patients with metastatic liver cancer, two with hepatocellular carcinoma and one with intrahepatic cholangiocarcinoma.

The pharmacokinetic analysis showed that patients were exposed only to low levels of MIV-818 and troxacitabine outside of the liver, providing experimental support for MIV-818’s liver targeting. The adverse events were mainly mild and the more serious side effects observed were reversible.

Biomarker analysis of liver biopsies from patients showed a selective liver cancer effect of MIV-818: while tumor tissue had clear DNA damage, healthy liver tissue showed only minimal or no DNA damage. Based on an independent expert analysis of liver tumor growth, five of the nine patients were assessed to have stable liver disease after treatment.

– The analysis of data from the nine patients confirms our conclusion from the first six patients and provides strong support for the continued development of MIV-818 as a new and effective treatment for liver cancer. MIV-818 is our most important project and therefore it feels very good that our confidence in the possibilities of the compound is further strengthened by the new data presented today, says Medivir’s CEO, Dr. Uli Hacksell.

The R&D Day will be streamed via a link on the website: www.medivir.com.

Medivir AB is obliged to make this information public pursuant to the EU Market Abuse Regulation.

The information was submitted for publication, through the agency of the contact person set out above, at 08.30 CET on 2 March, 2020.

About MIV-818

MIV-818 is a pro-drug designed to selectively treat liver cancers and to minimize side effects. It has the potential to become the first liver-targeted, orally administered drug to benefit patients with HCC and other forms of liver cancer.

On March 2, 2020 Neon Therapeutics, Inc. (Nasdaq: NTGN) reported financial results for the fourth quarter and full-year ended December 31, 2019 and provided a business update (Press release, Neon Therapeutics, MAR 2, 2020, View Source [SID1234555024]).

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"Earlier this year, we were delighted to announce our entry into a definitive merger agreement with BioNTech. Once closed, the transaction will combine two organizations with a common culture of pioneering translational science and a shared vision for the future of cancer immunotherapy," said Hugh O’Dowd, Chief Executive Officer of Neon. "We continue to be encouraged by the potential of our novel neoantigen-targeted T cell therapies and are confident that this merger with BioNTech will provide the foundation from which to ensure that potential will be realized."

BioNTech Transaction Details

Under the terms of the definitive merger agreement, Neon will, following consummation of the acquisition, merge with Endor Lights, Inc., a Delaware corporation and a direct, wholly-owned subsidiary of BioNTech SE (Nasdaq: BNTX) and become a wholly-owned subsidiary of BioNTech. At closing, BioNTech will issue, and Neon shareholders will receive, 0.063 American Depositary Shares (ADS) (each ADS representing one ordinary share of BioNTech) in exchange for each of their shares of Neon. The exchange ratio implies a deal value of approximately $67 million, or $2.18 per share of Neon, based on the closing price of BioNTech’s ADSs of $34.55 on Wednesday, January 15, 2020, the date of the definitive merger agreement.

The transaction was unanimously approved by both BioNTech’s and Neon’s boards of directors. The transaction, which is expected to close during the second quarter of 2020, is subject to approval by Neon’s shareholders and the satisfaction of customary closing conditions. Certain stockholders of Neon owning approximately 36% of the outstanding Neon shares have entered into voting agreements, pursuant to which they have agreed, among other things, and subject to the terms and conditions of the agreements, to vote in favor of the Neon acquisition.

Fourth Quarter and Full Year 2019 Financial Results:

Cash Position: As of December 31, 2019, cash and cash equivalents were $29.4 million, as compared to cash, cash equivalents and marketable securities of $103.3 million as of December 31, 2018.

R&D Expenses: R&D expenses were $12.7 million for the fourth quarter of 2019 and $59.7 million for the year ended December 31, 2019, as compared to $18.0 million for the fourth quarter of 2018 and $60.4 million for the year ended December 31, 2018. The decrease for both fourth quarter and full year 2019 was primarily driven by decreased costs supporting NEO-PV-01, including clinical development and manufacturing costs, as Neon shifted its strategic focus towards personal and precision neoantigen-targeted T cell therapy candidates. The decreases were partially offset by an increase in costs related to Neon’s investment in NEO-PTC-01 and other pre-clinical candidates that are aligned with Neon’s refined strategic focus, as well as an increase in personnel-related costs, including one-time restructuring charges in connection with our November 2019 workforce reduction.

G&A Expenses: G&A expenses were $5.3 million for the fourth quarter of 2019 and $21.4 million for the year ended December 31, 2019, as compared to $5.8 million for the fourth quarter of 2018 and $18.3 million for the year ended December 31, 2018. The decrease for the fourth quarter was primarily due to the timing of expenditures associated with protecting Neon’s owned and in-licensed intellectual property. The increase for

the full year was primarily due to increased personnel-related costs and costs associated with being a public company.

Net Loss Attributable to Common Stockholders: Net loss was $17.8 million for the fourth quarter of 2019 and $79.8 million for the year ended December 31, 2019, or a net loss per basic and diluted share of $(0.63) and $(2.86), respectively, as compared to a net loss of $23.1 million for the fourth quarter of 2018 and $83.3 million for the year ended December 31, 2018, or a net loss per basic and diluted share of $(0.84) and $(5.54), respectively.

On March 2, 2020 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an oncology-focused pharmaceutical company, reported the commencement of a registered underwritten public offering of $150 million in shares of its common stock (Press release, Karyopharm, MAR 2, 2020, View Source [SID1234555052]). In addition, Karyopharm also intends to grant the underwriters a 30-day option to purchase up to $22.5 million in shares of its common stock.

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J.P. Morgan, Morgan Stanley and Jefferies are acting as joint book-running managers for the offering.

An automatically effective shelf registration statement relating to the shares of common stock offered in the public offering described above was filed with the Securities and Exchange Commission (SEC) on February 26, 2020. The securities may be offered only by means of a written prospectus, including a prospectus supplement, forming a part of the effective registration statement. A preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. When available, copies of the preliminary prospectus supplement and the accompanying prospectus relating to the offering may also be obtained from J.P. Morgan Securities LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (866) 803-9204, or by email at [email protected]; Morgan Stanley & Co. LLC, 180 Varick Street, 2nd Floor, New York, NY 10014, Attention: Prospectus Department; or Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by email at [email protected], or by phone at (877) 821-7388.

This press release does not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On March 2, 2020 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported that management will present at the following investor conferences (Press release, Sangamo Therapeutics, MAR 2, 2020, View Source [SID1234555068]):

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Cowen 40th Annual Health Care Conference
Presentation Date: Wednesday, March 4th at 10:00 a.m. Eastern Time
Location: Boston, MA
Barclays Global Healthcare Conference
Presentation Date: Wednesday, March 11th at 8:30 a.m. Eastern Time
Location: Miami, FL
Presentations will be webcast live and may be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. The presentation will also be available on the Sangamo website after the event.